JOP20210231A1 - مركبات ترابطية مستهدفة كمركبات علاجية - Google Patents

مركبات ترابطية مستهدفة كمركبات علاجية

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Publication number
JOP20210231A1
JOP20210231A1 JOP/2021/0231A JOP20210231A JOP20210231A1 JO P20210231 A1 JOP20210231 A1 JO P20210231A1 JO P20210231 A JOP20210231 A JO P20210231A JO P20210231 A1 JOP20210231 A1 JO P20210231A1
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Jordan
Prior art keywords
targeting ligands
expression
compounds
target
inhibiting oligomeric
Prior art date
Application number
JOP/2021/0231A
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English (en)
Inventor
Pei Tao
B Rozema David
H Wakefield Darren
V Blokhin Andrei
D Benson Jonathan
Li Zhen
Fleitz Fred
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Arrowhead Pharmaceuticals Inc
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of JOP20210231A1 publication Critical patent/JOP20210231A1/ar

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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

يتعلق الاختراع الحالي بالكشف عن مركبات ترابطية مستهدفة جديدة يمكن ربطها بمركبات، هذة المركبات العلاجية التى تكون مفيدة في توجيه المركبات إلى داخل الجسم الحي المستهدف. يمكن أن تعمل المركبات الترابطية المستهدفة التي تم الكشف عنها في هذه الوثيقة على استهداف مركبات أوليجومرية لتثبيط التعبير، مثل عوامل ANAi، إلى خلايا الكبد لتضمين التعبير الجيني. يمكن استخدام المركبات الترابطية المستهدفة التي تم الكشف عنها في هذه الوثيقة، عند ترافقها مع مركب أوليجومرية لتثبيط التعبير في مجموعة متنوعة من التطبيقات التي تتضمن الاستخدام في تطبيقات علاجية، تشخيصية، التحقق من الهدف، وتطبيقات اكتشاف مجموعة العوامل الوراثية. تكون التركيبات التي تتضمن المركبات الترابطية المستهدفة التي تم الكشف عنها في هذه الوثيقة عند ربطها بمركبات أوليجومرية لتثبيط التعبير قادرة على التوسط فى التعبير عن متواليات حمض نووي مستهدفة في خلايا الكبد، مثل الخلايا الكبدية، التي يمكن أن تكون مفيدة في معالجة الأمراض أو الحالات التي تستجيب لتثبيط التعبير الجيني أو النشاط في خلية، نسيج أو عضو.
JOP/2021/0231A 2016-03-07 2021-08-19 مركبات ترابطية مستهدفة كمركبات علاجية JOP20210231A1 (ar)

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US201662304652P 2016-03-07 2016-03-07
US201662370754P 2016-08-04 2016-08-04
US201662426916P 2016-11-28 2016-11-28

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JOP/2017/0057A JOP20170057B1 (ar) 2016-03-07 2017-03-07 مركبات ترابطية مستهدفة كمركبات علاجية
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US (3) US10246709B2 (ar)
EP (1) EP3426261A4 (ar)
JP (1) JP6983797B2 (ar)
KR (3) KR102348425B1 (ar)
CN (2) CN113797348A (ar)
AU (2) AU2017229469B2 (ar)
BR (1) BR112018068230B1 (ar)
CA (1) CA3011946A1 (ar)
IL (3) IL296377A (ar)
JO (2) JOP20170057B1 (ar)
MX (3) MX2018009854A (ar)
TW (2) TWI727009B (ar)
UY (1) UY37146A (ar)
WO (1) WO2017156012A1 (ar)

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